SfN18: The potential of gene therapy in neurological disorders

Promising findings from preclinical animal studies presented at SfN Neuroscience (3–7 November, San Diego, CA, USA) show the potential of gene therapy in slowing down disease progression, and improving symptoms, in neurological disorders such as amyotrophic lateral sclerosis (ALS) and Parkinson’s disease (PD).

Although gene therapy is a promising treatment option for a limited number of conditions, the technique is still experimental for most diseases, with ongoing research to ensure they will be safe and effective in human patients.

New technique allows gene therapy to be delivered to the spinal cord in mice

A new technique for gene therapy can permeate the entire spinal cord and parts of the brain in a mouse model of ALS, according to researchers. To treat a disease like ALS with gene therapy, scientists hypothesize that it is necessary to repress the activity of the mutated SOD1 gene in cells throughout the entire CNS. However, current techniques are not very effective at delivering such silencing vectors through the spinal cord in adult animals.

M Bravo Hernandez (University of California San Diego, CA, USA) and colleagues developed a new technique for gene therapy delivery, in which a virus carrying a SOD1-silencing vector is injected underneath the thin membrane surrounding the spinal cord. This ‘subpial’ gene-delivery technique has been reported to be highly effective in delivering the vector to the entire length of the spinal cord, as well as brain cells that control voluntary motor function in adult mice, rats, pigs and non-human primates.

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